EP4087878A4 - Complexes de ciblage musculaire et utilisations de ces derniers pour le traitement de dystrophinopathies - Google Patents

Complexes de ciblage musculaire et utilisations de ces derniers pour le traitement de dystrophinopathies Download PDF

Info

Publication number: EP4087878A4
Authority: EP; European Patent Office
Prior art keywords: dystrophinopathies; treatment; muscle targeting; targeting complexes; complexes
Prior art date: 2020-01-10
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.): Pending

Application number

EP21738534.3A

Other languages

German (de)

English (en)

Other versions

EP4087878A1 (fr

Inventor

Romesh R. SUBRAMANIAN

Mohammed T. QATANANI

Timothy Weeden

Cody A. DESJARDINS

Brendan QUINN

Jason P. RHODES

Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)

Dyne Therapeutics Inc

Original Assignee

Dyne Therapeutics Inc

Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)

2020-01-10

Filing date

2021-01-08

Publication date

2024-06-05

2021-01-08 Application filed by Dyne Therapeutics Inc filed Critical Dyne Therapeutics Inc

2022-11-16 Publication of EP4087878A1 publication Critical patent/EP4087878A1/fr

2024-06-05 Publication of EP4087878A4 publication Critical patent/EP4087878A4/fr

Status Pending legal-status Critical Current

Classifications

- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
- A61K47/6849—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
- A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
- A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6889—Conjugates wherein the antibody being the modifying agent and wherein the linker, binder or spacer confers particular properties to the conjugates, e.g. peptidic enzyme-labile linkers or acid-labile linkers, providing for an acid-labile immuno conjugate wherein the drug may be released from its antibody conjugated part in an acidic, e.g. tumoural or environment
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
- C07K16/2881—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
- C07K2317/92—Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing

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Health & Medical Sciences (AREA)
Engineering & Computer Science (AREA)
Life Sciences & Earth Sciences (AREA)
Bioinformatics & Cheminformatics (AREA)
Chemical & Material Sciences (AREA)
Genetics & Genomics (AREA)
General Health & Medical Sciences (AREA)
Organic Chemistry (AREA)
Molecular Biology (AREA)
Medicinal Chemistry (AREA)
Biomedical Technology (AREA)
Pharmacology & Pharmacy (AREA)
Public Health (AREA)
Animal Behavior & Ethology (AREA)
Veterinary Medicine (AREA)
Immunology (AREA)
Biochemistry (AREA)
Biotechnology (AREA)
General Engineering & Computer Science (AREA)
Wood Science & Technology (AREA)
Zoology (AREA)
Epidemiology (AREA)
Biophysics (AREA)
Microbiology (AREA)
Plant Pathology (AREA)
Physics & Mathematics (AREA)
Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
Neurology (AREA)
Cell Biology (AREA)
General Chemical & Material Sciences (AREA)
Chemical Kinetics & Catalysis (AREA)
Physical Education & Sports Medicine (AREA)
Orthopedic Medicine & Surgery (AREA)
Proteomics, Peptides & Aminoacids (AREA)
Virology (AREA)
Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Peptides Or Proteins (AREA)
Medicinal Preparation (AREA)
Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

EP21738534.3A 2020-01-10 2021-01-08 Complexes de ciblage musculaire et utilisations de ces derniers pour le traitement de dystrophinopathies Pending EP4087878A4 (fr)

Applications Claiming Priority (8)

Application Number	Priority Date	Filing Date	Title
US202062959796P	2020-01-10	2020-01-10
US202062965748P	2020-01-24	2020-01-24
US202062968443P	2020-01-31	2020-01-31
US202062980874P	2020-02-24	2020-02-24
US202063055537P	2020-07-23	2020-07-23
US202063069066P	2020-08-23	2020-08-23
US202063132929P	2020-12-31	2020-12-31
PCT/US2021/012756 WO2021142307A1 (fr)	2020-01-10	2021-01-08	Complexes de ciblage musculaire et utilisations de ces derniers pour le traitement de dystrophinopathies

Publications (2)

Publication Number	Publication Date
EP4087878A1 EP4087878A1 (fr)	2022-11-16
EP4087878A4 true EP4087878A4 (fr)	2024-06-05

Family

ID=76788858

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
EP21738534.3A Pending EP4087878A4 (fr)	2020-01-10	2021-01-08	Complexes de ciblage musculaire et utilisations de ces derniers pour le traitement de dystrophinopathies

Country Status (10)

Country	Link
US (1)	US20230111212A1 (fr)
EP (1)	EP4087878A4 (fr)
JP (1)	JP7780691B2 (fr)
KR (1)	KR20220125802A (fr)
CN (1)	CN115335401A (fr)
AU (1)	AU2021205346A1 (fr)
BR (1)	BR112022013572A2 (fr)
IL (1)	IL294478A (fr)
MX (1)	MX2022008540A (fr)
WO (1)	WO2021142307A1 (fr)

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US11168141B2 (en)	2018-08-02	2021-11-09	Dyne Therapeutics, Inc.	Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20210308273A1 (en)	2018-08-02	2021-10-07	Dyne Therapeutics, Inc.	Muscle targeting complexes and uses thereof for treating dystrophinopathies
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JP2023535073A (ja) *	2020-07-23	2023-08-15	ダインセラピューティクス，インコーポレーテッド	ジストロフィン異常症を処置するための筋標的化複合体およびそれらの使用
AU2022298028A1 (en)	2021-06-23	2023-12-21	National Center Of Neurology And Psychiatry	Combination of antisense oligomers
US11771776B2 (en)	2021-07-09	2023-10-03	Dyne Therapeutics, Inc.	Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP4366784A1 (fr)	2021-07-09	2024-05-15	Dyne Therapeutics, Inc.	Complexes ciblant le muscle et formulations de traitement de dystrophinopathies
US11638761B2 (en)	2021-07-09	2023-05-02	Dyne Therapeutics, Inc.	Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11633498B2 (en)	2021-07-09	2023-04-25	Dyne Therapeutics, Inc.	Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11969475B2 (en)	2021-07-09	2024-04-30	Dyne Therapeutics, Inc.	Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11648318B2 (en)	2021-07-09	2023-05-16	Dyne Therapeutics, Inc.	Anti-transferrin receptor (TFR) antibody and uses thereof
JPWO2023026994A1 (fr) *	2021-08-21	2023-03-02
MX2024003373A (es) *	2021-09-16	2024-04-04	Dyne Therapeutics Inc	Dosis de complejos dirigidos a musculos para el tratamiento de distrofinopatias.
KR20250004770A (ko)	2022-04-15	2025-01-08	다인 세라퓨틱스, 인크.	근긴장성 이영양증을 치료하기 위한 근육 표적화 복합체 및 제제
WO2024097644A1 (fr) *	2022-10-31	2024-05-10	Dyne Therapeutics, Inc.	Complexes de ciblage musculaire et leurs utilisations pour sauter l'exon 45 d'un gène dmd
WO2024226471A2 (fr)	2023-04-24	2024-10-31	Biomarin Pharmaceutical Inc.	Compositions et méthodes de traitement de troubles liés à stxbp1
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2021
- 2021-01-08 EP EP21738534.3A patent/EP4087878A4/fr active Pending
- 2021-01-08 BR BR112022013572A patent/BR112022013572A2/pt unknown
- 2021-01-08 MX MX2022008540A patent/MX2022008540A/es unknown
- 2021-01-08 US US17/791,701 patent/US20230111212A1/en active Pending
- 2021-01-08 AU AU2021205346A patent/AU2021205346A1/en active Pending
- 2021-01-08 CN CN202180019467.9A patent/CN115335401A/zh active Pending
- 2021-01-08 IL IL294478A patent/IL294478A/en unknown
- 2021-01-08 JP JP2022542336A patent/JP7780691B2/ja active Active
- 2021-01-08 WO PCT/US2021/012756 patent/WO2021142307A1/fr not_active Ceased
- 2021-01-08 KR KR1020227027138A patent/KR20220125802A/ko active Pending

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AU2021205346A1 (en)	2022-09-01
KR20220125802A (ko)	2022-09-14
WO2021142307A1 (fr)	2021-07-15
US20230111212A1 (en)	2023-04-13
EP4087878A1 (fr)	2022-11-16
BR112022013572A2 (pt)	2022-09-13
CN115335401A (zh)	2022-11-11
IL294478A (en)	2022-09-01
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